title: Lipid-modified aminoglycoside derivatives for in vivo siRNA delivery.
abstract: Rationally designed siRNA delivery materials that are enabled by lipid-modified aminoglycosides are demonstrated. Leading materials identified are able to self-assemble with siRNA into well-defined nanoparticles and induce efficient gene knockdown both in vitro and in vivo. Histology studies and liver function tests reveal that no apparent toxicity is caused by these nanoparticles at doses over two orders of magnitude.
authors.affiliation: David H. Koch Institute for Integrative, Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA; Department of Anesthesiology, Children's Hospital Boston, Boston, MA 02115, USA.
authors.forename: Yunlong
authors.initials: Y
authors.lastname: Zhang
authors.forename: Jeisa M
authors.initials: JM
authors.lastname: Pelet
authors.forename: Daniel A
authors.initials: DA
authors.lastname: Heller
authors.forename: Yizhou
authors.initials: Y
authors.lastname: Dong
authors.forename: Delai
authors.initials: D
authors.lastname: Chen
authors.forename: Zhen
authors.initials: Z
authors.lastname: Gu
authors.forename: Brian J
authors.initials: BJ
authors.lastname: Joseph
authors.forename: Jasmine
authors.initials: J
authors.lastname: Wallas
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Anderson
mesh_terms: D000617:Aminoglycosides / Q000737:chemistry*; D000818:Animals; D018014:Gene Transfer Techniques; D006367:HeLa Cells; D006801:Humans; D008055:Lipids / Q000737:chemistry*; D051379:Mice; D034741:RNA, Small Interfering / Q000737:chemistry / Q000235:genetics / Q000378:metabolism*
chemical_list: D000617:Aminoglycosides; D008055:Lipids; D034741:RNA, Small Interfering
keywords: aminoglycoside; drug delivery; gene knockdown; nanoparticle; siRNA